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Wednesday, March 5, 2014

With Success, Pure-Play RNAi Therapeutics No More

As RNAi Therapeutics are charging forward towards first marketing approvals, it is time to consider how this will change the face of some of the pure-play companies involved. Alnylam is set to become the dominant player
in TTR amyloidosis, Arrowhead Research and Tekmira are vying to become just that in Hepatitis B, and Tekmira is increasingly isolating their
biodefense division involving the formation of a filovirus alliance with
non-RNAi modalities (prophylactic and therapeutic vaccines) as revealed yesterday.

Under normal circumstances, getting drugs approved that
revolutionize the treatment of particular diseases provides great incentives to
protect the resultant franchises.
Witness BiogenIdec . BiogenIdec started
out as a molecular biology pioneer and serendipitously established itself as the major player in
multiple sclerosis, and is now considering all therapeutic
modalities, including RNA Therapeutics, to build on that strength and further expand into neurology.

Although it breaks my scientific heart, I do not expect that to be any
different for RNAi Therapeutics companies.
HBV is a great example since an immune de-repressing RNAi Therapeutic
alone is unlikely to facilitate the 100% cure rates now seen with combinatorial
HCV treatments. Instead, the addition of
a direct immune-activating agent such as a TLR agonist should enhance cure rates
and shorten treatment durations. For an
acute fatal infection such as Ebola, you also do not want to limit yourself to
one treatment, but recruit multiple mechanisms to save an infected person, or
start vaccinating populations around areas of viral outbreaks.

To extract maximal value from such disease indications, it
is often best to combine the various options under one roof. It may be partly this fear of moving away
from your roots that is responsible for ISIS Pharmaceuticals giving up
marketing rights early on for a few cents on the dollar worth (SMN, myotonic
dystrophy, TTR).

Having said that, just like BiogenIdec with recombinant
proteins and monoclonal antibodies, most of these RNAi Therapeutics companies
will continue to focus their internal R&D efforts on RNAi Therapeutics. In special cases such as Ebola and other biodefense opportunities, a spin-out may create most shareholder value without damaging the
platform technology value of the parent company.

Nothing of what I just said is revolutionary. It is, however, worth keeping in mind as the
first major RNA(i) Therapeutics wave will make landfall in 2-3 years.

6 comments:

Anonymous
said...

The valuation gap between TKMR and ARWR and, in particular, DRNA seems quite strange. Is TKMRs tech really that inferior despite long history and considerable experience (and clinical/pre-clinical proof) and, in addition, the possibility of significant Ebola revenue in the next 2years...

No it's not, or course. TKMR mgmt needs to realize that in a game where cost of capital is crucial, they should not just cater to (potential) pharmaceutical collaborators, but also to the capital markets. They are a bit behind the curve here for sure. Still, I invest in the comfort of their scientific strength.

Is Benitec really going to be OK like everyone says it will be? That means Dirk is the one who has it all wrong. In which case, what are you going to do once you've been hung out to dry and the keyboard taken off of you?

What a petty and out of place comment. Why don't you wait for the science to prove itself before you say something. I doubt you'll show here to apologize and take your medicine if things don't go your way. So take the cheap shot and go home or say something on topic instead.

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